Ontology highlight
ABSTRACT:
SUBMITTER: Schoch KM
PROVIDER: S-EPMC5821515 | biostudies-literature | 2017 Jun
REPOSITORIES: biostudies-literature
Schoch Kathleen M KM Miller Timothy M TM
Neuron 20170601 6
Multiple neurodegenerative diseases are characterized by single-protein dysfunction and aggregation. Treatment strategies for these diseases have often targeted downstream pathways to ameliorate consequences of protein dysfunction; however, targeting the source of that dysfunction, the affected protein itself, seems most judicious to achieve a highly effective therapeutic outcome. Antisense oligonucleotides (ASOs) are small sequences of DNA able to target RNA transcripts, resulting in reduced or ...[more]